Sunday, February 8, 2026

Syndromic and Metabolic Cardiomyopathies: Rare Variants for Adults (Glycogen Storage Diseases, Friedreich’s Ataxia)

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Turk Kardiyol Dern Ars 2024;52(Suppl 1):S1-S206. In Friedreich’s Ataxia patients, concentric left ventricular hypertrophy is common; asymmet...
Tuesday, February 3, 2026

Clinical manifestations of Friedreich ataxia in patient with neurofibromatosis seen at Daoud Charity Clinic Sudan: A case report

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Abbasher Hussein, Mohajer Ismael, Mohamed Baraka, Abdaleliah Abobker, Wadah Altoom, Osama Suliman, Clinical manifestations of Friedreich ata...

Mitochondrial iron overload is associated with lysosomal dysfunction-mediated mitophagy impairment in the heart of Friedreich’s ataxia

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Eunbin Jee, Maisha Medha, Hwayoung Baek, Jonghan Kim, Yuho Kim, Mitochondrial iron overload is associated with lysosomal dysfunction-mediate...
Monday, February 2, 2026

La omaveloxolona recibe luz verde en España como primer tratamiento específico para la Ataxia de Friedreich

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Comisión Interministerial de Precios de los Medicamentos 28 de enero de 2026. Skyclarys H* (omaveloxolona): Tratamiento de la ataxia de Frie...
Friday, January 30, 2026

Larimar’s Pediatric Friedreich’s Ataxia Trial Termination: What Investors Should Know

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TipRanks Clinical-Trials-Jan 28, 2026  "The study was first submitted in November 2024, signaling the formal start of the regulatory pr...
Wednesday, January 14, 2026

California grants $7.4 million to advance gene-edited stem cell therapy for Friedreich’ s ataxia

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Gen 13, 2026. The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San D...

Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

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CHARLESTOWN, Mass., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. today announced that U.S. Food and Drug Administration (FDA) ha...
Monday, January 12, 2026

Participant in First-in-Class Phase 1b FALCON Trial Evaluating SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

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CHARLESTOWN, Mass., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. today announced that the first participant has been dosed in FA...
Saturday, January 10, 2026

Biogen Advances New SKYCLARYS Formulation in Early Trial, Supporting Long-Term Rare Disease Strategy

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TipRanks Clinical-Trials-Auto-Generated Newsdesk, Jan 09, 2026. Study Overview This Phase 1 Biogen study, officially titled “A Phase 1, Rand...
Friday, January 9, 2026

Insights into DNA repeat expansions among 900,000 biobank participants

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Hujoel, M.L.A., Handsaker, R.E., Tang, D. et al. Insights into DNA repeat expansions among 900,000 biobank participants. Nature (2026). doi:...